EMA reports surge in orphan drug applications in 2014
There were 329 applications, up from 201 in 2013
The latest annual report from the European Medicines Agency (EMA) has highlighted a major increase in applications for orphan drugs for rare diseases, which increased by 63% in 2014 year-on-year. There were 329 such applications last year; 201 in 2013; 197 in 2012; and 166 in 2011. Of these, 196 received positive opinions, compared with 136 in 2012.
The 2014 record was the highest number of orphan designations granted since the EU orphan medicinal products regulation (EC) No 141/2000 came into force in 2000.
EMA's annual report said the largest proportion by medicine type (29%) of these were for oncology products. The Agency gave 17 orphan-designated final marketing authorisations last year, also the highest number ever.
'As it is often not profitable for companies to develop medicines for rare diseases, EMA provides incentives to the developers,' said the report.
EMA provides incentives to the developers of medicines for rare diseases
In terms of final market authorisations for all types of human medicines, 82 were approved in 2014, although only one in two medicines that were granted a positive opinion contained a new active substance. There were 81 such approvals in 2013 and 57 in 2012.
'Developers of medicines are making more and better use of EMA’s tools aimed at helping patients to get access to effective and safe medicines more quickly,' said the report.
Meanwhile, with post-authorisation monitoring an increasingly important priority, the number of side effects in human medicines rose by 6.5% in 2014 compared with 2013.
The annual report also highlighted some EMA projects, initiatives and achievements in 2014, such as the new policy on clinical data publication.